BioCryst Pharmaceuticals, Inc. (Nasdaq: BCRX) today announced
new analyses of real-world use of oral, once-daily ORLADEYO®
(berotralstat) leading to a reduction in monthly attack rates in
patients with hereditary angioedema (HAE) who have normal
C1-inhibitor (C1-INH) level and function.
“There is a significant unmet need among people
who live with HAE with normal C1-INH, and identifying optimal
treatments has been challenging for these patients. These
real-world observations suggest ORLADEYO can have a meaningful
impact on the lives of people who have HAE with normal C1-INH. We
look forward to continuing to evaluate our oral, once-daily
prophylaxis as a treatment option for this subpopulation,” said Dr.
Ryan Arnold, chief medical officer of BioCryst.
The company also announced a new post-hoc
analysis from the APeX-S clinical trial that showed a sustained
reduction in HAE attacks compared to patients’ self-reported
baseline attack rates.
“The ability to compare patients’ treatment
outcomes with their HAE attack rates at baseline is tremendously
helpful to characterize the impact of a prophylactic therapy. While
this analysis from APeX-S includes baseline attack rates that were
retrospectively self-reported by patients, the findings are
consistent with results previously reported from the pivotal APeX-2
trial, that long-term prophylaxis with ORLADEYO leads to a
sustained reduction in attack rates and is an important therapeutic
option for the prevention of HAE attacks,” said H. James Wedner,
M.D., professor of medicine in the division of allergy and
immunology at the John T. Milliken department of medicine at
Washington University School of Medicine.
The data are being presented at the 2023 Annual Scientific
Meeting of the American College of Allergy, Asthma & Immunology
(ACAAI), which is being held at the Anaheim Convention Center in
Anaheim, Calif., from November 9-13, 2023.
BioCryst ACAAI 2023 Presentation Highlights
The presentations at ACAAI include analyses from
the APeX-S clinical study and real-world data from patients taking
ORLADEYO in the United States. APeX-S was a Phase 2, open label,
international study evaluating the safety and effectiveness of
ORLADEYO 110 mg once daily (QD) and 150 mg QD in patients with HAE
Type I or Type II for up to 96 weeks in the US and 240 weeks in all
other countries. The real-world data are analyses from
patient-reported results collected in the real-world clinical
setting from BioCryst’s sole-source pharmacy.
- Berotralstat Reduced Attack
Rates in Patients with Hereditary Angioedema with Normal
C1-Inhibitor: Real-World Outcomes; ePoster #P082; Friday,
November 10, 5:30-5:45 p.m. PT; Monitor #13, Exhibit Hall
- This analysis assessed
patient-reported HAE attack rates for patients with healthcare
provider-diagnosed HAE (reflective of an ICD-10 code of D84.1 or
T78.3) who have normal C1-INH level and function in the United
States and actively received ORLADEYO 110 mg or 150 mg QD at any
timepoint between December 16, 2020 and June 15, 2023 (n=302), with
data shown for up to 540 days. Data are also reported for a subset
of these patients who reported a 90-day baseline attack rate and
received ORLADEYO for ≥360 days (n=103). A sizeable number of
patients who received another prophylactic treatment for HAE at any
time, such as lanadelumab, intravenous and subcutaneous C1-INH and
androgens, were included in both cohorts.
- Patient-reported attack rates were
collected by the sole-source pharmacy at baseline and at each
refill (approximately every 30 days). The baseline 30-day average
was calculated based on each patient’s self-reported attack rate
for the 90 days prior to initiating ORLADEYO and by dividing that
value by three. Monthly attack rates were calculated by taking the
average of the reported attacks across each 90-day period.
- A reduction in HAE attack rates was
observed in both cohorts upon initiation of ORLADEYO:
- At baseline, the median attack rate
was 3.00 attacks per month (n=249). Upon initiation of ORLADEYO,
median attack rates were reduced to 1.00 at Days 1-90 (n=277) and
Days 91-180 (n=232); 1.29 at Days 181-270 (n=174); 1.00 at Days
271-360 (n=143); and 1.50 at Days 361-450 (n=105) and Days 451-540
(n=79), with a median attack rate of ≤1.50 attacks per month across
all reporting periods over the entire duration.
- For patients who reported a 90-day
baseline attack rate and received ORLADEYO for ≥360 days (n=103),
the median baseline attack rate was 3.00 attacks per month. Upon
initiation of ORLADEYO, median attack rates were reduced to 1.29 at
Days 1-90 (n=100); 1.00 at Days 91-180 (n=99); 1.33 at Days 181-270
(n=99); and 1.00 at Days 271-360 (n=101), with a median attack rate
of ≤1.33 attacks per month across all reporting periods over the
entire duration.
- This analysis suggests that
long-term prophylaxis with ORLADEYO resulted in a reduction in
patient-reported monthly attack rates compared to baseline and
median patient-reported attack rates remained consistently low in
patients with HAE who have normal C1-INH level and function.
- Berotralstat Reduced Attack
Rates Compared to Baseline in Patients with Hereditary Angioedema
in APeX-S; ePoster #P064; Saturday, November 11,
12:05-12:20 p.m. PT; Monitor #12, Exhibit Hall
- This analysis characterized the
safety and effectiveness of ORLADEYO 150 mg QD in U.S. patients
enrolled in the APeX-S trial who self-reported a baseline attack
rate (n=147). Patients were asked to recall the average number of
HAE attacks per month (attack rates) they experienced over the six
months prior to beginning therapy. Attack rates after beginning
therapy were calculated for each patient based on the number of
attacks they experienced that met predefined criteria, as specified
in the study protocol, and were adjusted for the duration of
treatment in each month. Attack rates at baseline were not
evaluated according to the predefined criteria used for inclusion
in the effectiveness analysis in APeX-S.
- ORLADEYO was generally well
tolerated, and safety was consistent with that of the entire APeX-S
population.
- In patients who reported HAE attack
rates at baseline, mean (SEM) attack rates declined from 2.3 (0.29)
self-reported attacks per month prior to initiating ORLADEYO
treatment to 0.71 (0.12) at Month 1, 0.49 (0.09) at Month 6 and
0.32 (0.10) at Month 12, respectively. A median attack rate of 0
attacks per month was observed for all months across the 12-month
period.
- Patients who were treated with
ORLADEYO experienced a sustained reduction in HAE attacks compared
to their self-reported baseline attack rates, suggesting a
reduction in disease burden and durable treatment effect.
In addition to being displayed in the exhibit
hall at the noted times, ePosters are accessible online and on
demand to registered attendees on ACAAI’s website.
About
ORLADEYO® (berotralstat)ORLADEYO® (berotralstat)
is the first and only oral therapy designed specifically to prevent
attacks of hereditary angioedema (HAE) in adult and pediatric
patients 12 years and older. One capsule of ORLADEYO per day works
to prevent HAE attacks by decreasing the activity of plasma
kallikrein.
U.S. Indication and Important Safety
Information
INDICATIONORLADEYO® (berotralstat) is a
plasma kallikrein inhibitor indicated for prophylaxis to prevent
attacks of hereditary angioedema (HAE) in adults and pediatric
patients 12 years and older.
Limitations of useThe safety
and effectiveness of ORLADEYO for the treatment of acute HAE
attacks have not been established. ORLADEYO should not be used for
the treatment of acute HAE attacks. Additional doses or dosages of
ORLADEYO higher than 150 mg once daily are not recommended due to
the potential for QT prolongation.
IMPORTANT SAFETY INFORMATION
An increase in QT prolongation was observed at
dosages higher than the recommended 150 mg once-daily dosage and
was concentration dependent.
The most common adverse reactions (≥10% and
higher than placebo) in patients receiving ORLADEYO were abdominal
pain, vomiting, diarrhea, back pain, and gastroesophageal reflux
disease.
A reduced dosage of 110 mg taken orally once
daily with food is recommended in patients with moderate or severe
hepatic impairment (Child-Pugh B or C) and in patients taking
chronically administered P-glycoprotein (P-gp) or breast cancer
resistance protein (BCRP) inhibitors (eg, cyclosporine).
Berotralstat is a substrate of P-gp and BCRP.
P-gp inducers (eg, rifampin, St. John’s wort) may decrease
berotralstat plasma concentration, leading to reduced efficacy of
ORLADEYO. The use of P-gp inducers is not recommended with
ORLADEYO.
ORLADEYO at a dose of 150 mg is a moderate
inhibitor of CYP2D6 and CYP3A4. For concomitant medications with a
narrow therapeutic index that are predominantly metabolized by
CYP2D6 or CYP3A4, appropriate monitoring and dose titration is
recommended. ORLADEYO at a dose of 300 mg is a P-gp inhibitor.
Appropriate monitoring and dose titration is recommended for P-gp
substrates (eg, digoxin) when coadministering with ORLADEYO.
The safety and effectiveness of ORLADEYO in
pediatric patients <12 years of age have not been
established.
There are insufficient data available to inform
drug-related risks with ORLADEYO use in pregnancy. There are no
data on the presence of berotralstat in human milk, its effects on
the breastfed infant, or its effects on milk production.
To report SUSPECTED ADVERSE REACTIONS,
contact BioCryst Pharmaceuticals, Inc. at 1-833-633-2279 or FDA at
1-800-FDA-1088
or www.fda.gov/medwatch.
Please see
full Prescribing
Information.
About BioCryst Pharmaceuticals
BioCryst Pharmaceuticals is a global biotechnology company with a
deep commitment to improving the lives of people living with
complement-mediated and other rare diseases. BioCryst leverages its
expertise in structure-guided drug design to develop first-in-class
or best-in-class oral small-molecule and protein therapeutics to
target difficult-to-treat diseases. BioCryst has commercialized
ORLADEYO® (berotralstat), the first oral, once-daily plasma
kallikrein inhibitor, and is advancing a pipeline of small-molecule
and protein therapies. For more information, please visit
www.biocryst.com or follow us on LinkedIn.
Forward-Looking Statements
This press release contains forward-looking
statements, including statements regarding future results,
performance or achievements. These statements involve known and
unknown risks, uncertainties and other factors which may cause
actual results, performance or achievements to be materially
different from any future results, performance or achievements
expressed or implied by the forward-looking statements. These
statements reflect our current views with respect to future events
and are based on assumptions and are subject to risks and
uncertainties. Given these uncertainties, you should not place
undue reliance on these forward-looking statements. Some of the
factors that could affect the forward-looking statements contained
herein include: the ongoing COVID-19 pandemic, which could create
challenges in all aspects of BioCryst’s business, including without
limitation delays, stoppages, difficulties and increased expenses
with respect to BioCryst’s and its partners’ development,
regulatory processes and supply chains, negatively impact
BioCryst’s ability to access the capital or credit markets to
finance its operations, or have the effect of heightening many of
the risks described below or in the documents BioCryst files
periodically with the Securities and Exchange Commission;
BioCryst’s ability to successfully implement its commercialization
plans for, and to commercialize, ORLADEYO, which could take longer
or be more expensive than planned; the commercial viability of
ORLADEYO, including its ability to achieve market acceptance; the
FDA or other applicable regulatory agency may require additional
studies beyond the studies planned for products and product
candidates, may not provide regulatory clearances which may result
in delay of planned clinical trials, may impose certain
restrictions, warnings, or other requirements on products and
product candidates, may impose a clinical hold with respect to
product candidates, or may withhold, delay, or withdraw market
approval for products and product candidates; BioCryst’s ability to
successfully manage its growth and compete effectively; risks
related to the international expansion of BioCryst’s business; and
actual financial results may not be consistent with expectations,
including that revenue, operating expenses and cash usage may not
be within management's expected ranges. Please refer to the
documents BioCryst files periodically with the Securities and
Exchange Commission, specifically BioCryst’s most recent Annual
Report on Form 10-K, Quarterly Reports on Form 10-Q, and Current
Reports on Form 8-K, which identify important factors that could
cause the actual results to differ materially from those contained
in BioCryst’s forward-looking statements.
BCRXW
Contact:John Bluth+1 919 859
7910jbluth@biocryst.com
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